pharmaphorum

JANUARY 9, 2023

Novartis’ programme providing free access to its spinal muscular atrophy (SMA) gene therapy Zolgensma is being scaled back to a dozen countries worldwide, according to the company. ” Zolgensma is one of the most expensive therapies available, with a price tag of around $2.1

Gene Therapy 119

Gene Therapy Genetics Gene Clinical Trials 119

pharmaphorum

NOVEMBER 19, 2021

Roche’s oral treatment Evrysdi for the rare genetic disease spinal muscular atrophy (SMA) will be made available on the NHS in England, after NICE reached a three-year access agreement with the company. million price tag make it is the most expensive treatment ever approved for NHS funding.

Drugs 97

Drugs Gene Therapy Genetic Disease Gene 97

pharmaphorum

JUNE 2, 2021

NICE has rejected routine NHS funding for Roche’s Evrysdi (risdiplam) for spinal muscular atrophy drug in draft guidance, a decision which doesn’t come as a surprise to patient association SMA UK. “NICE’s initial ‘no’ to approving risdiplam for NHS funding is not unexpected,” said SMA UK this morning.

Gene Therapy 52

Gene Therapy Gene Drugs Medicine 52

XTalks

OCTOBER 26, 2021

The treatment came with an initial price tag of $39,000 per vial, which amounts to a total of $575,00 each year. NICE provides health guidelines based on evidence-based assessments of efficacy and safety, and also serves as England’s drug price watchdog. The treatment will be offered through the UK’s National Health Service (NHS).

Gene Silencing 98

Gene Silencing Gene Clinical Trials Clinical Trials 98

pharmaphorum

DECEMBER 23, 2022

price tag of $475,000 when it was first launched in 2017 – and it becomes apparent that these may not be desirable treatment options for every patient and in every setting. Results suggest durable epigenetic re-wiring and provide rationale for intermittent clinical dosing. Professional reception of the findings.

Development 52

Development Drugs Clinical Development Antibody 52